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First evidence that CRISPR can edit genes directly inside the body
From From Bacterial Defence to Medicine: The CRISPR Gene-Editing Revolution
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Breakthrough
First evidence that CRISPR can edit genes directly inside the body
Intellia and Regeneron reported that a single intravenous infusion of NTLA-2001, a CRISPR therapy delivered by lipid nanoparticles, cut disease-causing TTR protein levels by up to 87% in patients with a rare amyloid disease.
Unlike Casgevy, which edits cells outside the body, NTLA-2001 is infused directly into the bloodstream, packaged in lipid nanoparticles that carry it to the liver, where it permanently switches off the gene producing a misfolded protein behind transthyretin amyloidosis. A single dose lowered serum TTR protein by up to 87% within weeks. It was the first published clinical evidence that CRISPR components could be delivered into a living patient and edit a target gene in place, without ever removing a single cell.
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